Trial Outcomes & Findings for A Pilot Clinical Trial With the Iron Chelator Deferiprone in Parkinson's Disease (NCT NCT01539837)

NCT ID: NCT01539837

Last Updated: 2020-06-16

Results Overview

To assess whether there were any serious adverse events in 6-month treatment with Deferiprone.

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 22 participants
• Primary outcome timeframe: 6 months
• Results posted on: 2020-06-16

Participant Flow

Participant milestones

Measure	Placebo Feriprox placebo administered orally at the same dosing volume as the 20mg/kg/day feriprox per day	Deferiprone 20mg 20mg/kg/day deferiprone divided into two equal doses (morning and evening), every day for 6 months	Deferiprone 30mg 30mg/kg/day Deferiprone divided into two equal doses (morning and evening), every day for 6 months
Overall Study STARTED	8	7	7
Overall Study COMPLETED	8	6	5
Overall Study NOT COMPLETED	0	1	2

Reasons for withdrawal

Measure	Placebo Feriprox placebo administered orally at the same dosing volume as the 20mg/kg/day feriprox per day	Deferiprone 20mg 20mg/kg/day deferiprone divided into two equal doses (morning and evening), every day for 6 months	Deferiprone 30mg 30mg/kg/day Deferiprone divided into two equal doses (morning and evening), every day for 6 months
Overall Study reduce neutrophil level	0	1	1
Overall Study bad compliance	0	0	1

Baseline Characteristics

A Pilot Clinical Trial With the Iron Chelator Deferiprone in Parkinson's Disease

Baseline characteristics by cohort

Measure	Placebo n=8 Participants Feriprox placebo administered orally at the same dosing volume as the 20mg/kg/day feriprox per day	Deferiprone 20mg n=7 Participants 20mg/kg/day deferiprone divided into two equal doses (morning and evening), every day for 6 months	Deferiprone 30mg n=7 Participants 30mg/kg/day Deferiprone divided into two equal doses (morning and evening), every day for 6 months	Total n=22 Participants Total of all reporting groups
Age, Continuous	64.38 years STANDARD_DEVIATION 3.23 • n=93 Participants	68.57 years STANDARD_DEVIATION 2.17 • n=4 Participants	62.85 years STANDARD_DEVIATION 2.74 • n=27 Participants	65.26 years STANDARD_DEVIATION 2.71 • n=483 Participants
Sex: Female, Male Female	5 Participants n=93 Participants	3 Participants n=4 Participants	2 Participants n=27 Participants	10 Participants n=483 Participants
Sex: Female, Male Male	3 Participants n=93 Participants	4 Participants n=4 Participants	5 Participants n=27 Participants	12 Participants n=483 Participants
Region of Enrollment United Kingdom	8 participants n=93 Participants	7 participants n=4 Participants	7 participants n=27 Participants	22 participants n=483 Participants
Parkinson's disease duration	3.54 years STANDARD_DEVIATION 0.34 • n=93 Participants	2.82 years STANDARD_DEVIATION 0.69 • n=4 Participants	3.02 years STANDARD_DEVIATION 2.69 • n=27 Participants	3.12 years STANDARD_DEVIATION 1.24 • n=483 Participants

PRIMARY outcome

Timeframe: 6 months

To assess whether there were any serious adverse events in 6-month treatment with Deferiprone.

Outcome measures

Measure	Placebo n=8 Participants Feriprox placebo administered orally at the same dosing volume as the 20mg/kg/day feriprox per day	Deferiprone 20mg n=7 Participants 20mg/kg/day deferiprone divided into two equal doses (morning and evening), every day for 6 months	Deferiprone 30mg n=7 Participants 30mg/kg/day Deferiprone divided into two equal doses (morning and evening), every day for 6 months
Number of Participants With Serious Adverse Events	0 Participants	0 Participants	0 Participants

SECONDARY outcome

Timeframe: 6 months

Assess whether Deferiprone therapy directly affects the symptoms of Parkinson's disease, modify regional brain mineralization (iron concentration) as assessed with T2* MRI in PD patients in the dentate nucleus. In previous animal studies, Deferiprone treatment reduced dentate nucleus iron content, as assessed by MRI. An increase in the T2*MRI value represents an increase in mineralization.

Outcome measures

Measure	Placebo n=8 Participants Feriprox placebo administered orally at the same dosing volume as the 20mg/kg/day feriprox per day	Deferiprone 20mg n=7 Participants 20mg/kg/day deferiprone divided into two equal doses (morning and evening), every day for 6 months	Deferiprone 30mg n=7 Participants 30mg/kg/day Deferiprone divided into two equal doses (morning and evening), every day for 6 months
Iron Concentrations in the Dentate Nucleus	30.74 ms Standard Error 0.65	30.59 ms Standard Error 0.87	29.86 ms Standard Error 1.10

Adverse Events

Placebo

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Deferiprone 20mg

Serious events: 0 serious events

Other events: 6 other events

Deaths: 0 deaths

Deferiprone 30mg

Serious events: 0 serious events

Other events: 6 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	Placebo n=8 participants at risk Feriprox placebo administered orally at the same dosing volume as the 20mg/kg/day feriprox per day	Deferiprone 20mg n=7 participants at risk 20mg/kg/day deferiprone divided into two equal doses (morning and evening), every day for 6 months	Deferiprone 30mg n=7 participants at risk 30mg/kg/day Deferiprone divided into two equal doses (morning and evening), every day for 6 months
Blood and lymphatic system disorders Decline in white cell counts	0.00% 0/8 • 6 months	14.3% 1/7 • Number of events 1 • 6 months	14.3% 1/7 • Number of events 1 • 6 months
Gastrointestinal disorders Gastrointestinal upset	0.00% 0/8 • 6 months	14.3% 1/7 • Number of events 1 • 6 months	28.6% 2/7 • Number of events 2 • 6 months
Musculoskeletal and connective tissue disorders Muscular joint pain	0.00% 0/8 • 6 months	57.1% 4/7 • Number of events 4 • 6 months	42.9% 3/7 • Number of events 3 • 6 months

Additional Information

David Dexter

Imperial College London

Phone: +44 (0)20 7594 6665

Email: d.dexter@imperial.ac.uk

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place